haptoglobin

The Phase I healthy trial revealed that NM8074 effectively blocked the alternative pathway (AP) and AP-mediated C3b deposition without affecting the classical pathway (CP). Importantly, NM8074 did not interfere with the deposition of C3b via the CP, a crucial marker of opsonization, across all cohorts in the Phase I trial.Regulatory approval for the Phase II trial in treatment-naïve PNH patients was granted based on the safety and tolerance demonstrated in the previous Phase I clinical trial involving healthy subjects. NM8074 proved to be safe and well-tolerated across various doses, ranging from 0.3 to 20 mg/kg.Subsequent dosing of NM8074 in treatment-naïve PNH patients yielded promising results. Notably, NM8074 led to significant reductions in Lactate Dehydrogenase (LDH), Reticulocytes, Free Hemoglobin, and D-dimer levels, while simultaneously increasing Hemoglobin and Haptoglobin levels.These results collectively suggest that NM8074 has the potential to selectively inhibit both intravascular hemolysis (IVH) and extravascular hemolysis (EVH) through the AP, while preserving the CP necessary for bacterial clearance and opsonization in treatment-naïve PNH patients. CLEVELAND, Oct. 30, 2023 (GLOBE NEWSWIRE) -- NovelMed Therapeutics is excited to announce the commencement of its Phase II clinical trial targeting treatment-naïve Paroxysmal Nocturnal Hemoglobinuria (PNH) patients with its groundbreaking anti-Bb antibody, NM8074. This open-label, multi-dose, and multi-center study aims to assess the safety and efficacy of NM8074 in PNH patient population. To date, eight PNH patients have been enrolled in the trial, with two patients already receiving NM8074 treatment. Key efficacy parameters include increases in Hemoglobin, increase in RBC clone size, decreased Lactate Dehydrogenase (LDH) levels, reduced pRBC transfusions, and reduced D-Dimer levels. Positive outcomes on these measures will pave the way for NM8074 as a highly differentiated and innovative biologic for treatment-naïve PNH patients. The Phase II study is divided into two cohorts, featuring a biweekly dosing schedule spanning a 3-month assessment period. Notably, the preliminary data indicates that NM8074 demonstrates a favorable safety profile, with no safety concerns. NM8074 was administered to treatment-naïve PNH patients with a clone size exceeding 10%. The results demonstrate significant reductions in LDH, Reticulocytes, Free Hemoglobin, and D-dimer, along with increases in Hemoglobin and Haptoglobin levels as expected. Furthermore, the clone size increased in a time-dependent manner. In summary, these observed changes in efficacy parameters validate the drug's mechanism of action and substantiate NM8074 as an effective PNH treatment option. Dr. Rekha Bansal, CEO of NovelMed Therapeutics, commented, "Preliminary data from the Phase II clinical trial in treatment-naïve PNH patients confirms NM8074's potential to comprehensively address intravascular and extravascular hemolysis-mediated anemia and bolster patient host defense systems, thus reducing the risk of infections." Previously, a Phase I trial involving forty healthy volunteers was successfully completed without any safety concerns. NM8074 was well tolerated across all doses, ranging from 0.3 to 20 mg/kg. These results corroborate NM8074's complete inhibition of the alternative pathway (AP) while preserving the classical pathway (CP), aligning with data generated by NovelMed's expert scientists. This selective mechanism distinguishes NM8074 from other complement blockers currently approved or in development by the FDA. About Paroxysmal Nocturnal Hemoglobinuria (PNH) PNH is a rare blood disorder characterized by the immune system's (Complement) attack on and destruction of PNH Red Blood Cells (RBC hemolysis). This leads to a decrease in hematocrit and hemoglobin levels, elevated LDH and D-Dimer, and an increased need for blood transfusions. PNH is a rare, chronic, and serious complement-mediated blood disorder with significant unmet needs. Despite the use of anti-C3 and anti-C5 therapies, many PNH patients continue to experience anemia, fatigue, and dependence on blood transfusions. About NM8074 NM8074 is a monoclonal antibody with dual specificity, differentiating it from other therapies such as Iptacopan, which binds to Factor B. NM8074 targets Bb and selectively blocks the alternative pathway (AP). This high-affinity monoclonal antibody inhibits C3b formation and deposition and membrane-attack complex (MAC) formation, offering a significant advantage in preventing the total lysis of red blood cells in PNH. Furthermore, NM8074 is being evaluated for other clinical indications, including atypical Hemolytic Uremic Syndrome (aHUS), for which it has received regulatory approval. NM8074's potential extends to complement-mediated pathologies in renal disorders, ocular disorders, neurological disorders, dermatological disorders, and inflammation, illustrating its broad application across multiple diseases. Dr. Rekha Bansal, Chief Executive Officer, emphasized, "This wide application to multiple diseases underscores our commitment to serving diverse patient populations." In contrast to other complement blockers from third parties, which indiscriminately block both the alternative and classical complement pathways, NM8074 offers a unique mechanism of action. It is expected to function as a superior therapeutic option, potentially without the Black-Box warning associated with currently FDA-approved treatments. About NovelMed Therapeutics NovelMed is a clinical-stage biopharmaceutical company dedicated to developing innovative biologics for the treatment of a wide range of complement-mediated diseases. NovelMed proudly leads in the development of anti-Bb antibodies to address chronic complement-mediated and complement-associated disorders. The company is actively conducting clinical trials in treatment-naïve PNH patients to expedite the availability of this promising treatment. Moreover, NovelMed is committed to delivering life-changing therapies to patients in the fields of hematology, ophthalmology, nephrology, dermatology, and neurology. NovelMed is Seeking Opportunities to Advance NM8074 for Rare Diseases. For more information, please visit https://www.novelmed.com/news Contact: Rekha BansalNovelMed Business Development Teambd@novelmed.com(216) 440 2696

A next generation human gut microbiome testing product range called MetaXplore™ has been launched in Australia to healthcare professionals. The MetaXplore™ range provides diagnostic gastrointestinal health testing together with metagenomic gut microbiome analysis. Following the launch in Australia, the product range is expected to be rolled out through Microba’s growing international distribution partner network. The MetaXplore™ test range is expected to expand the total addressable market for Microba’s testing products. BRISBANE, Australia--(BUSINESS WIRE)-- Microba Life Sciences Limited(ASX: MAP) (“Microba”), a precision microbiome company, is pleased to announce that it’s next generation testing product range, MetaXplore™ has been launched to healthcare professionals in Australia under a new brand, Co-Biome™. This delivers on the new healthcare product launch milestone prefaced in the FY22 Annual Report and updated in quarterly reports following the closed beta release in June 2022. This press release features multimedia. View the full release here: MetaXplore™ GI Plus can help you explore the impact your patients’ gut microbiome may be having on their health. MetaXplore™ GI Plus translates diagnostic gastrointestinal markers and targeted pathogen, protist and parasite panels1 alongside metagenomic-driven gut microbiome insights2 for over 28,000* microbial species and their function. (Photo: Business Wire) Microba’s Chief Executive Officer, Dr Luke Reid said, “The new MetaXplore™ test range delivers an important advancement in how microbiome test information can be applied in a healthcare setting. We continue to make progress at the forefront of this field to advance the application of microbiome testing in a healthcare setting towards a future where it can be routine in health and disease management, much like a blood test is today.” The new MetaXplore™ range provides diagnostic gastrointestinal health testing together with metagenomic-driven gut microbiome analysis to deliver comprehensive gastrointestinal testing solutions for Healthcare Professionals. The MetaXplore™ test range has been developed together with healthcare professionals and brought to market under a new brand called Co-Biome™. The range consists of 3 products: MetaXplore™, MetaXplore™ GI and MetaXplore™ GI Plus. Key features include: - Easy to interpret report format designed specifically for healthcare professionals to support efficient patient consultation - Scientifically graded clinical insights using National Health and Medical Research Council guidelines - Diagnostic and investigative gastrointestinal health markers (faecal calprotectin, faecal occult blood, lactoferrin, pancreatic elastase, secretory IgA, Zonulin, Fecal pH) - Diagnostic pathogen and parasite detection (E.coli pathotypes, C.difficile pathotypes, Campylobacter spp., Yersinia enterocolitica, Vibrio spp., Aeromonas spp., Salmonella spp., Giardia lamblia, Entamoeba histolytica, Cryptosporidium spp., Dientamoeba fragilis, Cyclospora cayetanensis.) - Complete microbiome profiling (Microbial diversity, richness and profiling of over 28,000+ microbial species including bacteria, fungi, parasite and archaea detection, together with parasite detection of Blastocystis subtypes 1-9 and other eukaryotes) - Microbiome production and consumption of metabolites (Butyrate, acetate, propionate, 3-indolepropionic acid (IPA), hexa-acylated, lipopolysaccharides (hexa-LPS), trimethylamine (TMA), hydrogen sulphide, branched-chain amino acids (BCAA), B.fragilis toxin, methane, beta-glucuronidase, Mucin, oxalate) A core component of Microba’s strategy is to leverage the Company’s world-leading technology and capability to advance the clinical application of microbiome testing to become embedded as a routine part of health and disease management. The MetaXplore™ test range through Co-Biome™ represents an important step forward in that strategy by breaking new ground in the application of microbiome testing in healthcare. Microba’s Chief Scientific Officer, Assoc Prof. Lutz Krause said, “These new tests integrate traditional gastrointestinal pathology tools with Microba’s next generation microbiome testing technology and the latest global research to significantly advance the utility of microbiome testing in a healthcare setting. This test range is expected to significantly expand the total addressable market for Microba’s testing products.” The test range through Co-Biome™ can be accessed in Australia through a healthcare professional, and following the launch, it is expected to then be rolled out through Microba’s growing international distribution partner network. About Microba Life Sciences Limited Microba Life Sciences is a precision microbiome company driven to improve human health. With world-leading technology for measuring the human gut microbiome, Microba is driving the discovery and development of novel therapeutics for major chronic diseases and delivering gut microbiome testing services globally to researchers, clinicians, and consumers. Through partnerships with leading organisations, Microba is powering the discovery of new relationships between the microbiome, health and disease for the development of new health solutions. View source version on businesswire.com: Contacts Simon Hinsley simon@nwrcommunications.com.au Source: Microba Life Sciences Limited Smart Multimedia Gallery Photo MetaXplore™ GI Plus can help you explore the impact your patients’ gut microbiome may be having on their health. MetaXplore™ GI Plus translates diagnostic gastrointestinal markers and targeted pathogen, protist and parasite panels1 alongside metagenomic-driven gut microbiome insights2 for over 28,000* microbial species and their function. (Photo: Business Wire) Photo The MetaXplore™ range provides healthcare professionals with a comprehensive view of diagnostic gastrointestinal markers and metagenomic-driven gut microbiome insights in an easy-to-interpret report. (Photo: Business Wire) Photo Discover the power of whole microbiome analysis and how deeper insights are addressing the way health professionals address gut health in practice. (Photo: Business Wire) Logo View this news release and multimedia online at:

Submission of NDA to the NMPA is planned within 2023 at the earliest SHANGHAI, Jan. 6, 2023 /PRNewswire/ -- VISEN Pharmaceuticals, an innovative biopharmaceutical company focused on endocrine diseases, announced today that it has completed the double-blind period of PaTHway China Trial, a phase 3 China clinical trial for its innovative investigational new prodrug palopegteriparatide on January 4, 2023, and it would start to carry out analysis of the primary endpoints. Palopegteriparatide is designed to restore parathyroid hormone (PTH [1-34]) to physiological levels over 24 hours in adult patients with hypoparathyroidism (HP). PaTHway China Trial is a randomized, double-blind, placebo-controlled, parallel, multi-center phase 3 trial to assess the potential of palopegteriparatide as a parathyroid hormone replacement therapy. The primary endpoints of the study include evaluating the safety, tolerability, and efficacy of once-daily subcutaneous injections of palopegteriparatide in adults with HP by measuring whether palopegteriparatide -treated patients achieve serum calcium (sCa) levels in the normal range (8.3–10.6 mg/dL) and independence from therapeutic levels of conventional calcium and active vitamin D therapy. VISEN obtained investigational new drug (IND) approval from the National Medical Products Administration (NMPA) on May 31, 2021, allowing it to initiate clinical research of palopegteriparatide in China. On June 16, 2022, VISEN completed patient enrollment for the PaTHway China Trial. HP is the last endocrine deficiency state, where hormone replacement so far has not been established as standard management. It is a rare endocrine disorder characterized by insufficient levels of parathyroid hormone (PTH), resulting in low calcium and elevated phosphate levels in the blood. Conventional therapy with calcium supplements and active vitamin D does not effectively address the short-term symptoms, long-term complications, or quality-of-life impacts of HP and can lead to impaired renal function. The once-daily subcutaneous injection of palopegteriparatide is designed to provide sustained exposure within normal physiological range of PTH concentrations. If approved, it is expected to become the first hormone replacement treatment targeting HP in China. Pony LU, CEO and Executive Director of VISEN Pharmaceuticals, said: "The completion of the double-blind period of the phase 3 clinical trial of palopegteriparatide in China means we have moved a step closer towards the success of our research efforts for what we hope will become China's first hormone replacement therapy targeting HP. We will now proceed with the analysis of the primary endpoints of the study and plan to submit a New Drug Application (NDA) to the National Medical Products Administration (NMPA) when all preparation work is completed. We look forward to bringing new treatment options to Chinese HP patients as soon as possible, so that their long-standing suffering could potentially be alleviated sooner." VISEN Pharmaceuticals is developing palopegteriparatide under license from Ascendis Pharma, who received Priority Review from the U.S. Food & Drug Administration (FDA) in 2022 for its New Drug Application (NDA) for this new drug. Ascendis has said that the U.S. FDA has set a target date for completion of its review of its application by April 30, 2023[1]. In addition, Ascendis Pharma also submitted a Marketing Authorization Application (MAA) for palopegteriparatide for adult patients with HP to the European Medicines Agency (EMA)[2]. [1] [2] SOURCE VISEN Pharmaceuticals